Galafold

Table of contents

Overview

This is a summary of the European public assessment report (EPAR) for Galafold. It explains how the Agency assessed the medicine to recommend its authorisation in the EU and its conditions of use. It is not intended to provide practical advice on how to use Galafold.

For practical information about using Galafold, patients should read the package leaflet or contact their doctor or pharmacist.

Galafold is a medicine used to treat patients aged 16 years or over with Fabry disease. This is a rare inherited disorder where patients have various mutations (changes) in the gene responsible for the production of an enzyme called alpha-galactosidase A, which normally breaks down a fatty substance called globotriaosylceramide (GL-3). In patients with Fabry disease, this enzyme does not work properly. As a result, GL-3 cannot be broken down and it builds up in various cells in the body, including in the heart and kidneys.

Because the number of patients with Fabry disease is low, the disease is considered ‘rare’ and Galafold was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 22 May 2006.

Galafold can only be obtained with a prescription and treatment should only be started and supervised by a doctor who is experienced in the diagnosis and treatment of Fabry disease.

Galafold is available as capsules (123 mg). The recommended dose of Galafold is one capsule every other day, taken by mouth at least 2 hours before or after food.

Galafold is only for use in patients with certain mutations in the alpha-galactosidase A gene. For more information, see the summary of product characteristics (also part of the EPAR).

The active substance in Galafold, migalastat, attaches to certain unstable forms of alpha-galactosidase A, stabilising the enzyme. This allows the enzyme to be transported into areas of the cell where it can break down GL-3.

Galafold has been investigated in two main studies involving a total of 127 patients with Fabry disease.

The first study, which compared Galafold with placebo (a dummy treatment) in 67 patients, looked at the proportion of patients who responded to treatment (defined as a reduction of at least 50% in GL‑3 deposits in the kidneys). Overall, Galafold was not found to be more effective than placebo at reducing GL‑3 deposits; however, additional analyses including only patients with those genetic mutations that can be treated with Galafold showed that patients responded better to Galafold than to placebo after 6 months of treatment.

The second study, in 60 patients, compared Galafold with the medicines agalsidase alfa or agalsidase beta, two treatments that replace the missing enzyme. The main measure of effectiveness was the change in patients’ kidney function after 18 months of treatment. In this study, Galafold was found to be as effective as enzyme replacement in stabilising patients’ kidney function.

The most common side effect with Galafold (which may affect around 1 in 10 people) is headache.

For the full list of all side effects and restrictions with Galafold, see the package leaflet.

The Agency’s Committee for Medicinal Products for Human Use (CHMP) decided that Galafold’s benefits are greater than its risks and recommended that it be approved for use in the EU. The Committee noted that Galafold was studied in a limited number of patients, however the available evidence is considered sufficient for such a rare disease. The CHMP also noted that Galafold is taken by mouth and this could be an advantage compared with other authorised treatments such as enzyme replacement which are given by infusion (drip) into a vein. Regarding safety, Galafold was well-tolerated.

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.

Orphan

This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Publication details

Marketing-authorisation holder

Amicus Therapeutics UK Ltd

Revision

7

Date of issue of marketing authorisation valid throughout the European Union

Pharmacotherapeutic group

migalastat

Therapeutic indication

Galafold is indicated for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation.